The median age at diagnosis of BCNS was 15 years, and most patients were diagnosed clinically.
Other abnormalities associated with BCNS were common: Eighty percent of patients had jaw cysts, 82% had palmer pitting, 50% had various bone abnormalities, 21% reported ovarian fibromas, 4% reported medulloblastomas, and 4% had other tumors.
and sporadic BCC, inactivating mutations and/or loss of PTCH1, or activating mutations of SMO, lead to constitutive activation of GLI transcription factors, expression of target genes, and cellular transformation.
This study was designed to assess the safety and efficacy of 150 mg of daily oral Erivedge versus placebo and enrolled 41 BCNS patients with surgically eligible BCCs from September 2009 to January 2011.
One of the main features of BCNS is development of multiple BCCs, including in areas that are not exposed to sunlight.
In binding to smoothened, the drug turns off the hedgehog-signaling pathway, which is inappropriately activated in patients with BCNS, leading to tumor cell growth and proliferation.
Tang and coworkers became interested in evaluating vismodegib in patients with BCNS after the drug was reported to be effective in a study involving 33 non-BCNS patients with locally aggressive or metastatic BCCs (N.
Remaining questions involving treatment of BCNS patients include the best vismodegib dosing regimen, and whether the tumors will eventually come back after the drug is stopped.
Michael Mena, Sales Director at BCNS
said: "Probably the best thing about working with GWAVA is the quality of their solutions.
This Phase II double blind, randomized placebo-controlled, two arm multicenter clinical study of vismodegib has enrolled forty-one BCNS patients from September 2009 to January 2011.
One of the prime features of BCNS is development of multiple BCCs, including in areas that are not exposed to sunlight.
Curis Cautionary Statement: This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including without limitation statements regarding: Curis's belief that this data provide compelling proof-of-concept for vismodegib in the treatment of BCC in BCNS patients.